Antibody-oligonucleotide conjugate compositions and methods for inducing dmd exon 45 skipping

Abstract

This publication discloses binding portion (e.g., antibody)-oligonucleotide (e.g., PMO) conjugates and pharmaceutical compositions that induce a mutation in a misspliced ​​dystrophin mRNA transcript to induce DMD exon 45 deletion. It also describes methods for treating muscular dystrophy, including Duchenne muscular dystrophy, comprising administering the conjugates or a pharmaceutical composition that induces a mutation in a misspliced ​​dystrophin mRNA transcript to induce DMD exon 45 deletion.